Say It Out Loud: There Is No Shame in Mental Illness. These are important words that every child, teen, and adult should hear! May is Mental Health Awareness Month, with…
According to a story from Rare Disease Report, a recent study indicates that the self-administration of Berinert, a drug used to treat hereditary angioedema, is effective and safe for patients…
According to a story from Rare Disease Report, the pharmaceutical company Alnylam Pharmaceuticals announced the results of Phase 3 trials for its experimental drug patisiran. The drug is in the…
National Nurses Week begins each year on May 6th and ends on May 12th, Florence Nightingale's birthday. Think about it... every time you visit a doctor's office or hospital, you…
According to a story from curetoday.com, the U.S. FDA has recently approved the CAR-T cell treatment Kymriah for adult patients with diffuse large B-cell lymphoma in cases of relapse or…
According to a story from Rare Revolution Magazine, Georgia Hart was first diagnosed with Friedrich's ataxia when she was 15 years old. Since then, living with this rare disease has…
Duchenne Muscular Dystrophy (DMD) is the most common type of the 9 types of muscular dystrophy. It is a genetic disorder that causes muscle degeneration and weakness. It is a progressive…
On February 13th, the U.S. FDA announced the approval of Symdeko, a novel combination of two previously used agents to treat cystic fibrosis (CF). Symdeko combines a drug that has been…
According to a story from BioSpace, the pharmaceutical company Scholar Rock recently announced that its drug candidate SRK-015 was recently granted Orphan Drug Designation by the U.S. FDA. The drug…
May the fourth be with you! This week we're spotlighting four patient stories: one about a father whose daughter has Sanfilippo syndrome, one about losing your spouse to CF, the…
According to a story from the Rare Disease Report, the results of a recent survey questionnaire indicate that communicating with other rare disease patients can cause a significant improvement in…
According to a story from Newswise, neurosurgeons at UC San Diego Health can now provide patients with an auditory brainstem implant (ABI) that can preserve some degree of hearing in…
A condition called XLHED that was thought to be incurable has been treated, and a paper describing the procedure, which can be found here, was released on the 26th April…
Narcolepsy is a rare disease subject to many misperceptions and false assumptions. All of that misinformation belies the fact that narcolepsy is a serious neurological disorder, with symptoms that can…
According to a story from bioportfolio.com, a recent study has demonstrated the effectiveness of Trelegy Ellipta, a unique three component combination treatment, in reducing the effects of chronic obstructive pulmonary…
Every year, May 20th is celebrated as International Clinical Trials Day. On this day organizations that are involved in the fields of science and medicine hold events to promote education…
Back in February, we covered the $1,000,000 IPF Catalyst Challenge. Announced in April, innovators from around the world submitted meaningful solutions to improve the quality of life of those living with…
According to a story from the Peterborough Telegraph, James Willis received a disaster diagnosis on his first Valentine's Day after getting married at age 29. James had been worried about…
According to a story from the Sydney Morning Herald, two-year-old William McLennan, son of Naomi Taylor and Ben McLennan, was diagnosed with spinal muscular atrophy about a year ago. The…
According to a story from news-medical.net, medical researchers have discovered that the gene called ari-1 can increase the risk aortic aneurysms. Previously, the precise genetic risk factors were not understood,…
The UK government is being petitioned to hold a referendum on the decriminalisation of the drug cannabis. This has implications for people with diseases that may be helped by using…
The National Center for Advancing Translational Sciences (NCATS) supports rare disease patients and their communities. They provide translational research funding, tools and other resources that are helping to address unique…
Click to read parts one and two of this story. According to the Center for Disease Control, Lyme disease is the fastest growing insect-borne infectious illness in the United States.…
Doctors haven’t been able to work out the cause of a young woman’s medical symptoms, and now the New York Times is appealing to the public for suggestions. This article…
Let's get moving in May! First off, for all you pop culture/Internet meme lovers, it's that time of year... May means we're on the eve of summertime, knee-deep in springtime,…
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